ClinicalTrials.gov
ClinicalTrials.gov Menu

The Dynamic Interplay Between Bleeding Phenotype and Baseline Factor Level in Moderate and Mild Hemophilia A and B (DYNAMO)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03623295
Recruitment Status : Recruiting
First Posted : August 9, 2018
Last Update Posted : August 9, 2018
Sponsor:
Information provided by (Responsible Party):
Karin Fijnvandraat, Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

Brief Summary:

There are large inter-individual differences in the bleeding pattern of patients with moderate or mild hemophilia. The major determinant of bleeding phenotype is the level of coagulant factor VIII or IX. In hemophilia A, studies addressing the association between factor VIII level and the clinical bleeding pattern yield conflicting results. In hemophilia B such studies have not yet been performed.

The primary aim of this project is to analyze the association between factor VIII and factor IX levels and the bleeding phenotype. The secondary aim is to analyze potential differences in phenotype between hemophilia A and B.

The project is a multicentre observational cohort study. We will include 500 patients with moderate or mild hemophilia A (FVIII 0.02-0.35 IU/mL) and 500 patients with moderate or mild hemophilia B (FIX 0.02-0.35 IU/mL) who are 12 to 55 years old. The main cohort study consists of clinical data collection, one blood sample and an online questionnaire for patients. Data will be collected on the nature and duration of all bleeding episodes, disease and treatment characteristics, physical activity level and musculoskeletal status. One blood withdrawal will be performed for centralized laboratory assays for FVIII or FIX levels (both one-stage and chromogenic assays) and genetic analysis for the most prevalent prothrombotic mutations. The online questionnaire for patients focuses on bleeds experienced in the past.

A subset of 200 patients aged 24 years or older (100 with moderate or mild hemophilia A and 100 with moderate or mild hemophilia B) will be investigated in more detail by longitudinal data collection including analysis of physical joint status, MRI imaging of joints and biomarkers for joint damage. This longitudinal observation will consist of two time points that lie two years apart, allowing us to identify any changes that occur over the observed time period with respect to joint status.


Condition or disease Intervention/treatment
Hemophilia Other: Blood sample Other: Questionnaire Other: MRI-imaging Other: Physical examination

Study Type : Observational
Estimated Enrollment : 1000 participants
Observational Model: Cohort
Time Perspective: Other
Official Title: The Dynamic Interplay Between Bleeding Phenotype and Baseline Factor Level in Moderate and Mild Hemophilia A and B
Actual Study Start Date : January 1, 2018
Estimated Primary Completion Date : April 1, 2020
Estimated Study Completion Date : April 1, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Bleeding Hemophilia

Group/Cohort Intervention/treatment
Cohort study population
For the main cohort study, we will include 500 patients with moderate or mild hemophilia A and 500 patients with moderate or mild hemophilia B.
Other: Blood sample
Blood withdrawal.

Other: Questionnaire
Online questionnaire about the bleeds that patients experienced in the past.

Sub study population
A subset of 200 patients of the cohort study population will be investigated in more detail by longitudinal data collection.
Other: Blood sample
Blood withdrawal.

Other: MRI-imaging
MRI imaging of joints.

Other: Physical examination
Physical examination of joint status.




Primary Outcome Measures :
  1. Bleeding phenotype [ Time Frame: Retrospective 10 years ]
    Annual bleeding rate, annual major bleeding rate, annual spontaneous joint bleeding rate, annual joint bleeding rate


Biospecimen Retention:   Samples With DNA

Biospecimens to be retained for future research:

  • Blood
  • Urine


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   12 Years to 55 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Patients will be recruited from Hemophilia Treatment Centers participating in the INSIGHT consortium, a well-established and productive group of investigators from European countries, Canada and Australia.
Criteria

Inclusion Criteria:

  • Moderate or mild hemophilia A (FVIII:C 0.02-0.35 IU/mL) or hemophilia B (FIX:C 0.02-0.35 IU/mL)
  • Age from 12 up to and including 55 years

Exclusion Criteria:

  • Other clotting disorder
  • Participation in another trial with an investigational product
  • Comorbidity affecting the musculoskeletal status
  • Clinically relevant inhibitor status at present or in the past
  • Hemophilia B Leyden
  • Use of anticoagulants

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03623295


Contacts
Contact: Anne-Fleur Zwagemaker +31205668668 a.zwagemaker@amc.nl
Contact: Fabienne Kloosterman +31205668668 f.r.kloosterman@amc.nl

Locations
Australia
Royal Adelaide Hospital Not yet recruiting
Adelaide, Australia
Contact: INSIGHT study team    +31205668668    insight@amc.nl   
Austria
Medical University of Vienna Not yet recruiting
Vienna, Austria
Contact: INSIGHT study team    +31205668668    insight@amc.nl   
Belgium
Multicentre: Leuven, Brussels Not yet recruiting
Multiple Locations, Belgium
Contact: INSIGHT study team    +31205668668    insight@amc.nl   
Canada
Multicentre: Vancouver, Toronto, Hamilton Not yet recruiting
Multiple Locations, Canada
Contact: INSIGHT study team    +31205668668    insight@amc.nl   
Finland
Helsinki University Central Hospital Not yet recruiting
Helsinki, Finland
Contact: INSIGHT study team    +31205668668    insight@amc.nl   
Germany
Multicentre: Bonn, Berlin, Frankfurt, München, Hamburg Not yet recruiting
Multiple Locations, Germany
Contact: INSIGHT study team    +31205668668    insight@amc.nl   
Italy
Multicentre: Florence, Rome, Parma, Milan, Turin Not yet recruiting
Multiple Locations, Italy
Contact: INSIGHT study team    +31205668668    insight@amc.nl   
Netherlands
Academic Medical Center Recruiting
Amsterdam, Netherlands
Contact: INSIGHT study team    +31205668668    insight@amc.nl   
University Medical Center Groningen Recruiting
Groningen, Netherlands
Contact: INSIGHT study team    +31205668668    insight@amc.nl   
Leiden University Medical Center Not yet recruiting
Leiden, Netherlands
Contact: INSIGHT study team    +31205668668    insight@amc.nl   
Maastricht University Medical Center Not yet recruiting
Maastricht, Netherlands
Contact: INSIGHT study team    +31205668668    insight@amc.nl   
Radboud University Medical Center Not yet recruiting
Nijmegen, Netherlands
Contact: INSIGHT study team    +31205668668    insight@amc.nl   
Erasmus Medical Center Not yet recruiting
Rotterdam, Netherlands
Contact: INSIGHT study team    +31205668668    insight@amc.nl   
Utrecht University Medical Center Not yet recruiting
Utrecht, Netherlands
Contact: INSIGHT study team    +31205668668    insight@amc.nl   
Máxima Medical Center Not yet recruiting
Veldhoven, Netherlands
Contact: INSIGHT study team    +31205668668    insight@amc.nl   
Spain
Multicentre: Valencia, Madrid, Barcelona Not yet recruiting
Multiple Locations, Spain
Contact: INSIGHT study team    +31205668668    insight@amc.nl   
United Kingdom
Multicentre: Manchester, London, Liverpool, Glasgow, Cardiff, Sheffield Not yet recruiting
Multiple Locations, United Kingdom
Contact: INSIGHT study team    +31205668668    insight@amc.nl   
Sponsors and Collaborators
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Investigators
Principal Investigator: Karin Fijnvandraat Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

Responsible Party: Karin Fijnvandraat, Professor of Pediatric Hematology, Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
ClinicalTrials.gov Identifier: NCT03623295     History of Changes
Other Study ID Numbers: NL61564.018.17
First Posted: August 9, 2018    Key Record Dates
Last Update Posted: August 9, 2018
Last Verified: August 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants